$30 Million Cash Infusion for San Diego Firm, On Top of $20 Million from California Stem Cell Agency

A recent recipient of nearly $20 million from the California stem cell agency is on a financial roll this week as it pursues its efforts to translate what it says are "best-in-class gene therapy technologies into lifesaving cell therapies."

The firm is Poseida Therapeutics, Inc., of San Diego. Last October, it was awarded $19.8 million by the California Institute of Regenerative Medicine (CIRM), as the stem cell agency is formally known.

Poseida said yesterday it has hauled in $30.5 million more. The firm said in a news release that the cash will go to "advance a pipeline of autologous and allogeneic CAR-T immunotherapies, as well as gene therapies, using Poseida’s suite of gene engineering technologies."

The firm said its CIRM-backed "P-BCMA-101(product) is a CAR-T therapy currently in Phase 1 clinical development for relapsed/refractory multiple myeloma, with initial clinical data accepted for presentation at the upcoming AACR (American Association for Cancer Research) Annual Meeting." The firm is also tackling prostate cancer.

Jack Murtha of Health Care Analytics News wrote,

"It’s a good day for Poseida Therapeutics, one of the trailblazing precision medicine ventures that aims to transform cancer treatment through cutting-edge gene-editing technologies. But just how good of a day is it? Try $30.5 million good."

Murtha said the company has now raised more than $80 million for its endeavors, including the CIRM award, venture capital of $11.2 million last August and $23 million in 2015. The bulk of today's funding came from Longitude Capital of Menlo Park.

At the time of the CIRM award last fall, Maria Millan, president of CIRM, described the nature of the problem that Poseida was addressing, 

“Multiple myeloma disproportionately affects people over the age of 65 and African Americans, and it leads to progressive bone destruction, severe anemia, infectious complications and kidney and heart damage from abnormal proteins produced by the malignant plasma cells. Less than half of patients with multiple myeloma live beyond 5 years.”

Last fall, CIRM reviewers, meeting behind closed doors, unanimously approved, 10-0, Poseida Therapeutics application (CLIN2-10395), which included Poseida's commitment to provide $8.6 million in co-funding. The agency's governing board later ratified the decision with no discussion.

A public, CIRM-prepared summary of the reviewers' comments said, 

"Reviewers thought that the proposed product has the potential to provide a very high rate of durable response in myeloma patients. There is strong scientific and clinical rationale for targeting BCMA on myeloma cells. Reviewers thought that the proposed improvements to the CAR T cell platform technology are highly innovative and could enhance the efficacy and durability of the treatment. Reviewers unanimously recommended the application for funding."

Job Openings at California's Stem Cell Agency

The California stem cell agency, which is set to basically expire in less than two years, is looking for a few good folks to continue to help it out.

The California Institute for Regenerative Medicine (CIRM), as the agency is formally known, has posted openings for an associate director of marketing communications and interns in the communications and human resources areas.

The associate director's responsibilities include oversight of the agency's Web site plus additional task. The person will replace Karen Ring, who is leaving to join Weber Shandwick, a major public relations firm. Here is the job information for for the marketing position.

The internships are unpaid and part-time. Here are links to the CIRM information on those openings: Communications,which also includes Web work, and human resources.

Currently the Oakland-based agency has a little more than 50 employees. It predicts it will run out of cash for new awards by the end of 2019 unless ambitious private fund-raising plans are successful.

New Tool for Wading Through California's $2.5 Billion in Stem Cell Spending

The $3 billion California stem cell agency this week added a valuable feature that is aimed at giving better access to information concerning its active research portfolio, which currently runs to more than 160 projects.

The feature is called the Active Awards Portfolio Dashboard. Its development was overseen by Karen Ring, the agency's social media honcho. Writing on the agency's blog, she said,

"This interactive tool makes it easy to search through the active research projects that we’re currently funding, and filter these projects by disease focus, technology type or stage of research."

The new dashboard is a companion to the clinical awards dashboard, also engineered by Ring. The agency's website contains vast quantities of information. The dashboards provide tools that help readers wade through it and ferret out what is most relevant to their individual needs.

The California Institute for Regenerative Medicine(CIRM), as the agency is formally known, has funded 969 projects costing nearly $2.5 billion during its 13-year history . It is slated to run out of cash in 2019 unless ambitious fundraising plans fulfill their promise. 

Parkinson's Patient Advocate Pitches for More California Stem Cell Funding

Sometimes things move quickly at meetings of the governing board of the California stem cell agency, which is formally known as the California Institute for Regenerative Medicine(CIRM).  At the meeting this month, it ended before a patient advocate had a chance to make her voice heard.

She is Jenifer Raub, president of the Summit for Stem Cell Foundation of San Diego. She requested that this web site carry the comments she would have made to the board. We are delighted to provide them below.

“On behalf of Summit for Stem Cell Foundation we applaud the efforts of CIRM to move Stem Cell Research forward. We task you specifically with addressing and funding a stem cell-based cell therapy for Parkinson’s Disease  (PD) Research. There is tremendous enthusiasm within the PD community for an autologous iPSC-based therapy. Parkinson’s Advocates are a vocal group as you well know. Funding assistance from CIRM for an autologous iPSC-based therapy for PD would easily result in Parkinson’s Advocates gratefully voicing support for funding for CIRM on the 2020 ballot.”

The California Stem Cell Report welcomes comments and lengthier commentary from persons interested in the $3 billion stem cell agency. Please send them to djensen@californiastemcellreport.com.

Stem Cell Blog Taking Week Off

The California Stem Cell Report is going dark this week. Look for exciting and thrilling fresh postings next week.

California's Alpha Clinic Stem Cell Program Shares Stories and Results Next Month

Brain cancer is the target of this Alpha Clinic effort 
at the City of Hope, which produced this video.

Come April 19th the topic at UCLA will be "Delivery of Stem Cell Therapeutics to Patients" and will feature much of the work being done at the Alpha Clinics that have been generated by the California stem cell agency.

The day-long program is free and open to the public. Registration is required. The session features researchers from throughout the state, ranging from Mehrdad Abedi of UC Davis to Joseph Ciacci of UC San Diego.

Last year's Alpha symposium was held at the City of Hope and was greeted by a packed house.

The Alpha Clinics are located at UC Davis, UC San Francisco, the City of Hope, UC San Diego and UCLA/Irvine and have been financed by the California Institute for Regenerative Medicine(CIRM), as the agency is formally known, at a cost of $50 million.

The clinics have engaged in 48 clinical trials, many of which draw funding from sources other than CIRM. The idea behind the Alpha program is to bring resources together to focus intensely on stem cell therapies and patient treatment.

The agency says the Alpha Clinics feature:

• "Patient-centered clinical trials with the highest standards of care and research
• "Strategic collaboration with academic and industry teams developing innovative stem cell therapies 
• "Leveraging of resources to promote efficiency, acceleration and increased chances of success"

UC Davis and UC San Francisco were added to the list of Alpha Clinics last September. 

Potential Death of California Stem Cell Program Hangs Over Its Performance Evaluation

The looming demise of California's $3 billion stem cell research effort dominated today's meeting of its governing board, which was told both that the agency has made "incredible progress" but needed to do better.

The occasion was the presentation of a $230,000 performance audit required by state law, a task performed by the firm of Moss Adams, one of the largest accounting and consulting firms in the world.

The firm's 38-page report targeted fund-raising, retention of staff and better utilization of board members in its recommendations for improvement and called for a more concrete plan for raising more than $200 million. Excerpts from the report can be found here and an earlier story on the audit findings here.

In its oral presentation, Moss Adams representatives had a number of good things to say about the stem cell agency, formally known as the California Institute for Regenerative Medicine (CIRM). They cited the agency's "incredible progress" and said "we usually see a lot of good things here."

It was the third go-around for Moss Adams, which has performed all three of the CIRM performance audits. (See this for links to the previous audits.) Today's presentation, however, focused more on the issues surrounding the agency's end within a few years, unless ambitious fundraising plans are successful.

The agency expects to run out of cash for new awards next year unless it is successful at raising $200 million from private contributors. That would take care of the agency's needs until a possible $5 billion bond measure might be approved by California voters in 2020.

One board member, Jeff Sheehy, also a member of the San Francisco board of supervisors, raised a number of critical questions, seeking more details about the fund-raising effort. He said there seemed to be a "lot of uncertainty" about it.

Jonathan Thomas, chairman of the agency, said a fund-raising plan is in place. He is leading the effort and being assisted by two other employees. He said some board members have been engaged and he plans to consult with all 29 board members.

David Higgins, a board member from San Diego, asked Thomas about a "double-edged sword" that could come into play because of a successful fund-raising effort. Higgins said the public might be more likely to oppose to a new bond measure if the $200 million is raised, reasoning that public funding was not needed in light of private support. Thomas said he did not think that was a major problem.

The Good and the Not-So-Good: Excerpts From an Evaluation of the California Stem Cell Agency

The latest performance audit of California's $3 billion stem cell agency reports both pluses and minuses on the agency's work. Here are some excerpts from the $230,000 study, the third such audit of the California Institute for Regenerative Medicine (CIRM).

"CIRM has a collaborative, engaged, and performance-oriented culture. Managers, Board members, and staff report improved morale and a more collaborative culture since CIRM 2.0 implementation. CIRM appointed a new president in 2014 and again in 2016. Turnover is often elevated during times of leadership transitions, as noted in 2014 and 2015. However, CIRM’s turnover rate dropped to 10 percent in 2016, suggesting stability in the organization’s leadership and culture."

"CIRM’s uncertain future funding results in employee concerns related to job security. These concerns can, in turn, translate into poor employee morale and reduced organizational productivity. Employees facing potential layoffs often experience elevated stress and anxiety that can become evident in their day-to-day interactions with colleagues and grantees. Because individual employee performance and team performance impact overall organizational performance, declining employee morale can negatively impact CIRM’s ability to efficiently and effectively achieve its mission."

"CIRM’s strategic plan emphasized that CIRM is patient-centered. As such, CIRM has increased its stakeholder engagement, particularly with patient advocates. The agency hosts patient events and trainings across the State of California and has at least 1,000 patient advocates registered. Additionally, CIRM’s Science Officers now proactively search for viable projects, helping engage researchers and increase the quality of proposed grants." 

"As CIRM’s Proposition 71 funding is exhausted, the role of the Communications Department should be elevated to serve a strategic function in educating the public. It is imperative that members of the public are aware of and understand CIRM’s activities and impact while CIRM remains fact-based and objective about its role as a state agency. To achieve this, CIRM should establish metrics focused on external communication and public awareness. Examples of these metrics include: Number of posts on social media accounts, number of press mentions, follower growth on social media accounts, number of 'likes' or 'shares' on social media accounts, growth in website visitors, citations of CIRM-funded research in publications These metrics provide insight on the organization’s communications output, reach, and engagement with audiences beyond patient advocates."

"With the implementation of CIRM 2.0, the organization improved many of its processes to be more efficient and effective. Examples of key process improvements include: ... Establishing “The Wall” by developing a policy clarifying the roles and responsibilities of CIRM employees within and outside the grant review process to ensure it remains fair and impartial.  Streamlined the review summary process by reducing the time to summarize grant applications from six weeks to approximately three weeks.  Reduced grant processing time by reducing the time required to process grants from 22 months to 120 days.  Incorporated milestone-based payments in contracts by shifting the responsibility for project progress to grantees and ultimately holding them responsible for achieving research objectives."

"The ICOC is a large, statewide governing board, with 29 members located across California. There are inherent challenges to this structure: Geography limits the ability of some members to participate in Agenda Item #8 ICOC Meeting March 13th, 2018 California Institute for Regenerative Medicine FY 2016-2017 Performance Audit  17 person and interface with CIRM staff; individuals on a large board may feel less personally responsible and therefore less inclined to participate; and it is difficult for officers and committee chairs to meaningfully build relationships with and identify the best roles for such a large number of members. This is evident in committee participation; while CIRM leverages ICOC expertise through standing and ad-hoc committees, which is a best practice, some members serve on multiple committees while others serve on none. In addition to these challenges, changes to CIRM’s conflict of interest policy in 2013 eliminated the ability of institutional members to participate in grant funding votes. This has limited their overall participation, and several ICOC members reported a reluctance to participate in discussions. Both ICOC members and CIRM staff reported a decline in the engagement of institutional members in all discussions, not just those related to grants."

"In its transition plan, CIRM leadership identified $200 million in additional required funding to continue operating at existing capacity between 2019 and the potential bond measure in 2020. This additional funding would enable the organization to maintain current annual grant awards and staffing levels. Currently, key board members are leading fundraising efforts, which have remained largely confidential. In order to secure this significant amount of funding, additional resources are likely required to demonstrate CIRM’s funding needs, seek donors of varying size and requirements, and provide overall support for fundraising efforts."

"Because the level of funding required to sustain CIRM’s operations is significant, CIRM should develop a formal fundraising plan to identify required resources, activities, and strategies. CIRM’s fundraising plan should identify additional resources and support within the organization required to help potential funders understand the benefit of their investment. For example, the role of the communications team could be expanded to develop fundraising materials that detail CIRM’s goals, plans, and successes as well as funding requirements."

"Transition plans and associated strategies should continue to be deployed as living documents that are continually updated and available to Board members, staff, and external stakeholders. Future iterations should also include organizational structure options during a potential wind-down process. For example, grant funds are currently expected to be exhausted by 2019; grant monitoring, management, clinical trials, and closeout will continue past that date; and intellectual property reporting will be required for 10 years. Eventually, some CIRM activities could be transferred to another state agency. As the transition proceeds, stakeholders and grantees should be made aware of staffing changes to help preserve continuity."

Performance Audit of California Stem Cell Agency: Improvements Needed in $200 Million Fund-Raising Program, Staff Retention and Use of Board Members

A performance audit of California's $3 billion stem cell agency says that more needs to be done to retain the agency's staff as well as exploiting the talents of  board members as the research effort nears what could be its demise.

"There is a significant potential for staff attrition as CIRM plans for a potential wind-down," the $230,000 study by Moss Adams declared. CIRM is the abbreviation for the agency's official name,nthe California Institute for Regenerative Medicine.

The 38-page report also said that expertise of  the 29 board members "could be more effectively leveraged, especially since strong board engagement will be particularly important during organizational transition."

CIRM has estimated it will run out of cash for new awards in 2019 unless ambitious fund-raising plans are successful.  Its ultimate hope is that California voters will approve another multi-billion dollar bond measure in November 2020. To bridge the financial gap between 2019 and the end of 2020, the agency is seeking to raise more than $200 million.

Loss of employees has long been a concern of the agency as it confronts the expiration of the bond funding provided by the ballot initiative that created the program in 2004.

The performance audit said the agency should be "building on efforts to date, continue to regularly communicate transition plans to staff and consider strategies to retain employees, including implementing staff development programs, recognition and reward opportunities, work-life balance initiatives, and cross-functional initiatives."

Beyond its recommendations, the audit had praise for the agency. One example involved its "culture." The report said,

"CIRM has a collaborative, engaged, and performance-oriented culture. Managers, board members, and staff report improved morale and a more collaborative culture since CIRM 2.0 implementation. CIRM appointed a new president in 2014 and again in 2016. Turnover is often elevated during times of leadership transitions, as noted in 2014 and 2015. However, CIRM’s turnover rate dropped to 10 percent in 2016, suggesting stability in the organization’s leadership and culture."

Moss Adams, however, also said more can be done. It said the agency should "develop succession plans for the chair and vice chair, document knowledge of individuals serving in leadership roles, and continue to identify potential highly qualified prospective (board) members."

The audit recommended improvements in the agency's fund-raising efforts. It said the agency has not "formalized its ($200 million) fund development plan." The audit recommended creation of "a formal development plan that identifies roles and responsibilities and the timing of fundraising activities to meet CIRM’s programmatic and administrative funding needs."

Moss Adams additionally recommended improvements in measuring the performance of the agency's public relations program. It said,

"CIRM does not have metrics associated with the effectiveness of the agency’s communication and public education strategy. Recommendation: Develop communications and public education metrics that are integrated into CIRM’s quarterly reporting."

The study, which is required by state law, is the third conducted by Moss Adams. It will be discussed at Tuesday's meeting of the agency's board. The agenda for that meeting said the agency will have a response to the audit findings, but that document is not yet available on the CIRM web site.

The audit, which is required by state law, is the agency's third. Links and excerpts from items involving the previous two can be found here. 

Interested parties can participate in the meeting at 16 different locations throughout California. The  locations can be found on the agenda, but more specifics may be necessary for those who want to attend. They can be had by sending an email to info@cirm.ca.gov.

California Stem Cell Performance Audits and More: Public Has 16 Golden Opportunities to Weigh In

Californians throughout the state next week will have what appears to be a record number of opportunities to participate -- albeit remotely -- in one of the more important, regular public meetings involving the $3 billion state stem cell agency.

The occasion is the March 13 meeting of the governing board of what is known formally as the California Institute for Regenerative Medicine or CIRM. The meetings of the CIRM governing board are the single most important public events that the agency holds.

The board has 29 members. Fifteen of those members will be participating remotely in next week's session, which means that the public can participate as well from those locations in addition to the physical site of the meeting at CIRM HQ in Oakland. The remote locations range from Fresno to La Jolla  and from Riverside to Elk Grove.

And if you are in New York City, you can weigh in from there as well. One of the agency directors will be hooked up from the Big Apple.

During the meeting, the board solicits public comment on each item under consideration plus general comments as well.

The top item on the agenda is the triennial performance audit of the agency, which in the past has

Graphic by Shopify

reported checkered but improving results in the last two reports. Conflict of interest issues surfaced in the last audit(see below).  In 2012, the audit, required by state law, identified 27 areas where improvements were needed.

The actual audit for this year has not yet been posted on the CIRM web site but is likely to pop up any day now.  The audit, which is required by state law, cost the agency $230,000, bringing to $694,944 the total that the agency has spent on its three performance audits.

If you are interested in attending at the remote locations, check the addresses in advance. Sometimes not enough specificity is provided. But an email to info@cirm.ca.gov will provide full directions.

Here is a link to the 2017 CIRM request for a proposal to conduct the performance audit.

Below are excerpts and links to previous items on the two earlier performance reports. At the end is a comparison of performance audit findings to results from other studies of the agency.

SUNDAY, MAY 17, 2015

Conflict-of-Interest Failings Reported in Application Reviews at California Stem Cell Agency

Conflict-of-interest issues have dogged the $3 billion California stem cell agency since its inception, and they are surfacing once again this week in a report commissioned by the agency itself.

This time the matter is being brought up by Moss-Adams, LLP, of Seattle, a business consulting firm that is being paid $230,000 by the agency to conduct a "performance audit."

In a report to be discussed at a CIRM governing board meeting on Thursday, the firm said it discovered serious problems dealing with the reporting of the interests of the agency's blue-ribbon reviewers.

TUESDAY, MAY 19, 2015

California Stem Cell Audit: Praise for Mills but More Work Needed on IP, Conflicts of Interest

The California stem cell agency this week received good marks for changes made by its new president, but it is also being told that it needs to improve how it tracks potential royalties and how it prevents grant reviewer conflicts of interest.

SUNDAY, MAY 17, 2015

Conflict-of-Interest Failings Reported in Application Reviews at California Stem Cell Agency

Conflict-of-interest issues have dogged the $3 billion California stem cell agency since its inception, and they are surfacing once again this week in a report commissioned by the agency itself.

This time the matter is being brought up by Moss-Adams, LLP, of Seattle, a business consulting firm that is being paid $230,000 by the agency to conduct a "performance audit."

In a report to be discussed at a CIRM governing board meeting on Thursday, the firm said it discovered serious problems dealing with the reporting of the interests of the agency's blue-ribbon reviewers.

THURSDAY, MAY 24, 2012

CIRM Directors Pleased with Performance Audit Findings

The $3 billion California stem cell agency received a "very favorable" performance audit report compared to other government agencies, CIRM directors were told today.

Representatives of Moss Adams, which was paid $234,944 by CIRM for the study, made the comments during a presentation today to the agency's 29 directors. During their comments, CIRM executives and directors focused on the favorable aspects of the findings of the six-month study.

TUESDAY, MAY 15, 2012

IP to Grant Oversight: Study Calls for Host of Improvements at California Stem Cell Agency

The $3 billion California stem cell agency is laboring under a range of problems that include protection of its intellectual property and management of its nearly 500 grants plus an inadequate ability to track its own performance, a seven-month study said yesterday.

The performance audit by the Moss Adams accounting firm of Seattle, Wash., made 27 recommendations for improvements, including more effort to ease strain connected to the agency's controversial dual executive arrangement. The study said that the nearly eight-year-old agency has many "opportunities" to "enhance performance reporting and decision making, strengthen effectiveness and efficiency, retain essential human resources and leverage technology."

In response to the report, the stem cell agency said, "(M)anagement concurs with the findings and recommendations....The recommendations are focused and constructive. CIRM is already implementing many of these recommendations, and we will be investigating the others in the coming months."

--

Here is a link to the California state controller's comparison of the findings of the 2012 performance audit to previous findings from a number of enterprises ranging from the National Academy of Sciences to the state's Little Hoover Commission. 

Aussie Bioethics Web Site Negative on California Stem Cell Royalties

The California stem cell agency and its royalty news drew attention today from down under in an article that did not exactly view the research effort with warm regards.

The piece appeared on the bioethics blog called Bioedge and was written by Michael Cook, who publishes the web site. He picked up on American news about the royalty matter, largely from the California Stem Cell Report, but also from the $3 billion agency itself.

Cook described the critics as scathing. He concluded,

"During the campaign for Proposition 71, supporters strongly argued that destructive research on human embryos was absolutely necessary for the science to proceed and that cures would certainly come. Almost 14 years later, there have been no cures. The royalty cheque was for a potential therapy for glioblastoma, a deadly brain tumour – but even this not on the market yet. Thus far, it has only passed Stage I clinical trials and been written up in glowing terms in O, The Oprah Magazine."